The licensed IP is based, in part, on the discovery of a researcher at Seoul National University and co-founder of ToolGen, which can be used to engineer cells found in mammals and plants.
CRISPR/Cas9 is a gene-editing system discovered in bacteria, which use it to spot and snip invading virus DNA.
“Our innovation allows researchers to cut at a specific site of the genome, which is required for efficient changing of genetic information in genomes. Our system was developed in 2012 and is composed of a protein component and an RNA component,” said Seokjoong Kim of ToolGen.
“We can programme the RNA component to target a specific site, which enables highly efficient genome editing compared to previous platforms like zinc finger nucleases and TALENS.”
There have been a variety of patent claims on CRISPR technologies, including by Cellectis, CRISPR Therapeutics, Editas and Intellia Therapeutics. Kim said ToolGen’s IP not only contains important core claims for the use of CRISPR technology for genome editing but also boasts a number of unique points, such as the “ways to modify the RNA for specificity” and being “the first to show that we can use the CRISPR/Cas system in enzyme form.”
“People had used a DNA- or RNA-based vector for the expression of Crispr/CAS9 system in cell, but we showed an enzyme, RNP complex of Crispr/CAS9, can be used in vivo and in vitro supporting efficient genome editing with improved specificity, which could provide interesting ways to apply this technology to cells or for developing therapies,” he explained.
Scientists have used CRISPR to replace genes or replace their DNA code and show it can be used to cure mice of some diseases like muscular dystrophy.
Regards patent disagreements, Kim said it is “hard to predict how the patent situation will be resolved,” but is confident that ToolGen’s patents have strong and unique claims.
“It will be hard to predict who will have what rights,” he said, but he points to a previous dispute over IP over TALENS. “Instead of actually fighting over it, those involved decided to create partnerships. This then enables users of the technology like pharmaceuticals to move in via licensing and apply the technology.”
He is hopeful that CRISPR will meet a similar denouement: “I think the patent issue with CRISPR will need to be solved but that the players won’t let this complex situation go on for too long.”
ToolGen will keep its rights in broad areas including screening, diagnostics, bioproduction and gene therapy. It will continue to focus on establishing global partnerships to maximise the potential of its technologies.
Helge Bastian, vice president and general manager of synthetic biology at Thermo, said: “Genome editing technologies are among the most exciting innovations in the life science in recent years, truly enabling scientists to understand gene function, the underlying molecular mechanisms for cell function and disease onset and progression.”
“This license agreement, as part of Thermo Fisher Scientific’s larger strategy, underlines Thermo Fisher Scientific’s commitment to invest into further developing these genome editing technologies into groundbreaking products that enable our customers to make our world healthier, cleaner and safer.”